Advice on a potential candidate for conditional inclusion of ataluren (Translarna®) for Duchenne's muscular dystrophy

Zorginstituut Nederland recommends that ataluren be designated as a potential candidate for conditional inclusion. Ataluren is registered for the treatment of Duchenne's muscular dystrophy due to a nonsense mutation in the dystrophin gene, in ambulatory patients aged two years and older.

Zorginstituut's advice

On 10 June 2020, the parties submitted an application for conditional inclusion in orphan drugs, conditionals and exceptionals for ataluren (Translarna®). Ataluren is registered for the treatment of Duchenne's muscular dystrophy due to a nonsense mutation in the dystrophin gene, in ambulatory patients aged two years and older.

Based on the data in the dossier and the advice of the Scientific Advisory Board (WAR), the Zorginstituut has concluded that treatment with ataluren in this group of patients with Duchenne's meets the criteria for conditional inclusion.

Based on the data it has collected, this study is expected to provide an answer to the question about inclusion in the Dutch insured package. The Zorginstituut anticipates that the question about inclusion in the Dutch insured package will be answered within 2.5 to 3 years.

Phase 2 of the procedure will commence when the Minister adopts this advice. We ask the parties to formulate their plans in greater detail and to draw up a covenant setting out the agreements needed to ensure that the CI process is conducted carefully and successfully. The Ministry of Health, Welfare and Sport will have to conclude a financial arrangement with the marketing authorisation holder. 

Conditional inclusion

Whether a form of treatment or a medicinal product is worth including in the basic health care package depends on the availability of clinically relevant evidence of its effectiveness. Such information is generally not available (in sufficient quantities) at the moment of registration of medicinal products for severe - often less prevalent - diseases.

Conditional inclusion in the package will allow marketing authorisation holders an opportunity to carry out further research into the effectiveness and appropriate use in the Netherlands of these medicinal products.

Based on the research outcomes, the Zorginstituut will eventually be able to issue advice on whether a medicinal product complies with ‘established medical science and medical practice’, which means that its effectiveness has been scientifically proven.

This report is a summary of recommendations by Zorginstituut Nederland. The original text is in Dutch.